Results 41 to 50 of about 31,863 (238)
Oman Journal of Ophthalmology, 2009 Hemophilic pseudotumor is an uncommon complication of factor VIII and IX deficiencies in the coagulation cascade and occurs in a wide spectrum of bones and soft tissues. We present a six-year-old boy with hemophilic pseudotumor localized in the right orbit.
Gunaseelan Karunanithi +3 more
openaire +3 more sources
Neuroprotection, EarlyView.Abstract Prime editing, a novel clustered regularly interspaced short palindromic repeats (CRISPR)‐based technology, fuses a reverse transcriptase (RT) to an engineered CRISPR‐associated protein 9 (Cas9) and uses a prime editing guide RNA (pegRNA)‐encoded template.
Tianshan Ji +4 more
wiley +1 more source
Hemophilia A: An Ideal Disease for Prenatal Therapy
Prenatal Diagnosis, EarlyView.ABSTRACT Hemophilia A (HA) is the most common inherited coagulation defect. Current state‐of‐the‐art treatment consists of frequent administration of prophylactic infusions of coagulation factor VIII (FVIII) protein or bispecific antibodies that replace the cofactor function of FVIIIa to maintain hemostasis. However, these treatments are far from ideal,
Christopher D. Porada +2 more
wiley +1 more source
The Milbank Quarterly, EarlyView.Policy Points The original purpose of the 340B program was to exempt Public Health Service Act funded clinics and state and local public hospitals from the inflationary best‐price component of the recently enacted Medicaid drug rebate program. The secondary purpose was to reduce drug prices for these clinics and hospitals in order to preserve and ...
SAYEH NIKPAY +2 more
wiley +1 more source
The association between unemployment and treatment among adults with hemophilia
Research and Practice in Thrombosis and HaemostasisBackground: People with hemophilia often experience pain and suffer from comorbidities related to their bleeding disorder. Consequently, unemployment due to disability is prevalent among people with hemophilia. Objectives: To explore associations between
Christian Qvigstad +27 more
doaj +1 more source
British Journal of Haematology, EarlyView.Gene therapy is revolutionizing treatment paradigms for haemoglobinopathies, establishing a translational framework for disorders that impact red blood cell development. In their paper, Joshi et al. describe the preclinical and early clinical landscape of gene therapies for non‐haemoglobinopathy erythroid disorders and highlight common thematic ...
Gaurav Joshi +3 more
wiley +1 more source
Determinants of Immune Tolerance Induction Outcomes in 237 Persons With Severe Hemophilia A
European Journal of Haematology, EarlyView.ABSTRACT Immune tolerance induction (ITI) to eradicate an immune response against factor VIII (FVIII) has been used in hemophilia A (HA) with various success rates, costs, and treatment burden. The present study aimed to identify markers predictive of ITI success.
Marcus Fager Ferrari +5 more
wiley +1 more source
Therapeutic Advances in HematologySummary What is this study about? Type 1 plasminogen deficiency (PLGD-1 for short) is a long-term, ultra-rare condition where the body does not make enough of a protein called plasminogen.
Amy D. Shapiro +7 more
doaj +1 more source
Emicizumab in the treatment of hemophilia A [PDF]
Acta Stomatologica Naissi, 2020 Introduction: Despite substantial advances in the treatment of Hemophilia A with the use of concentrated factor VIII preparations during recent decades, bleeding episodes still occur from time to time.
Tijanić Ivan R. +3 more
doaj
European Journal of Haematology, EarlyView.ABSTRACT Introduction Central venous catheter (CVC) insertion is frequently required in patients with haematological diseases. Despite limited evidence, thrombocytopenia often prompts prophylactic platelet transfusion before catheterisation. Methods We conducted an observational before‐and‐after study including the first non‐tunnelled CVC insertion ...
Mathias Lazarevic Lindblad +8 more
wiley +1 more source